Conference Day One
Wednesday, August 21

8:30 am Morning Check-In

8:55 am Chair’s Opening Remarks

Navigating Target Specificity Through Engineered Delivery Systems for Enhanced Therapeutic Impact

9:00 am Panel Discussion: Beyond the Liver: Unleashing the Potential of Next Gen Lipid-Based Therapies

Synopsis

  • Expanding the scope of lipid-based therapies beyond the liver to address diverse disease targets throughout the body
  • Exploring novel delivery systems and formulations that harness lipid-based technologies for treating extrahepatic disorders with enhanced precision
  • Pioneering the era of personalized medicine by tailoring lipid-based therapies to individual patient profiles, revolutionizing treatment approaches for various medical conditions

9:30 am Harnessing Targeting Moieties for Success in Targeted Drug Delivery

Synopsis

  • Explore the criteria and considerations for identifying cell receptors and targeting moiety modality for effective targeting within lipid nanoparticles
  • Discuss how the unique properties of antibodies contribute to improved precision and efficacy in drug delivery
  • Highlight innovative techniques and breakthroughs that overcome integration challenges while preserving the biological activity of targeting moieties

10:00 am Targeting LNPs With Small Molecule Ligands & Antibody Fragment

Synopsis

  • Active targeting strategies for LNPs
  • Balancing potency and selectivity
  • Use of DC-targeted LNPs in development of a highly effective M. Tuberculosis vaccine

10:30 am Morning Refreshment & Speed Networking

11:30 am Stability Engineering in LNPs for Robust Delivery

  • Ching Kim Tye Head Of Lipid Nanoparticle Platform Development, Sanofi

Synopsis

  • Strategies for reinforcing lipid nanoparticle stability through molecular engineering, ensuring structural integrity and preventing premature payload release
  • Developing LNPs with enhanced resistance to environmental challenges, such as changes in pH, temperature, and enzymatic degradation, to maintain stability during the complex journey from administration to target site
  • Addressing challenges in extending the shelf life of LNPs through innovative formulation techniques and storage protocols, ensuring sustained stability for effective and reliable drug delivery

12:00 pm Proprietary Ionizable Lipids-Based LNP for Muscle-Selective Gene Delivery With Minimized Systemic Exposure via Local Administration

Synopsis

  • Developing novel ionizable lipids for next-generation of LNP for muscle-selective gene delivery
  • Enhancing locally restricted gene delivery with a notable reduction in undesired systemic exposure
  • Enabling the development of mRNA vaccines for infectious diseases and cancer with reduced adverse events

12:30 pm Advancing Pulmonary Delivery of Therapeutic mRNA

  • Sandy Liu Sr Scientist, Omega Therapeutics, Inc.

Synopsis

  • Explore innovative techniques to improve the precision of pulmonary delivery, ensuring therapeutic mRNA reaches its intended target within the lungs
  • Discuss strategies to navigate the complex pulmonary environment, including mucus barriers and immune responses, to optimize mRNA delivery and efficacy
  • Learn about cutting-edge advancements in pulmonary delivery technology, paving the way for transformative treatments in respiratory diseases and beyond

1:00 pm Lunch Break & Networking

Turbocharging Non-Clinical Development for Precision in Off-Target Effect Reduction

2:00 pm Roundtable Discussion: Addressing Challenges in Animal Models for Translatable LNP Development

Synopsis

  • Unraveling the discrepancies between animal models and human responses in LNP delivery, confronting the challenges of species-specific variations in physiology and metabolism
  • Developing innovative strategies to enhance the predictive value of animal models, acknowledging limitations and implementing adjustments for a more accurate reflection of human LNP delivery dynamics
  • Exploring the incorporation of human-relevant elements in preclinical studies, from utilizing humanized models to embracing in vitro systems, to foster more reliable predictions of LNP behavior and efficacy in clinical settings

3:00 pm Phospholipids in Vaccines

Synopsis

  • The amphiphilic nature and safety profile of phospholipids make them ideal excipients for LNP formulations
  • The lipid composition of LNPs and their impact on the delivery of mRNA
  • Helper lipid selection can modulate the properties of LNP delivery and stability
  • DSPC with its high phase transition temperature provides excellent colloidal stability to LNPs
  • DOPE can enhance endosomal escape, while other phospholipids can target specific tissues
  • Phospholipids and their role in delivering adjuvanted liposomal vaccines

3:30 pm Afternoon Break & Poster Session

4:00 pm Advancing Gene Therapy: Navigating Formulation Challenges & LNP Manufacturing Impact

Synopsis

  • Formulation consideration for non-viral gene delivery
  • LNP manufacturing: Impact on physical attributes and functionality

4:30 pm Roundtable Discussion: Cracking the Code – Payload Challenges in Engineering LNPs for Precision Delivery

Synopsis

  • Navigating the intricate landscape of payload diversity within LNPs, exploring the challenges posed by different therapeutic agents and the need for tailored engineering solutions to accommodate varied chemical and physical properties
  • Unraveling the complexities of achieving high loading efficiency while ensuring payload stability, addressing the intricacies associated with encapsulating diverse payloads, from small molecules to nucleic acids
  • Discussing the challenge of balancing stability and controlled release within the dynamic and heterogeneous biological environments, emphasizing the need for nuanced LNP engineering strategies to optimize therapeutic outcomes

5:00 pm Chair’s Closing Remarks

5:15 pm Close of Conference Day One